Study: Existing blood pressure drug may slow rare childhood brain disease: Study | Morong Express

Representative image (Source: Xinhua/IANS)

The Hague, August 16 An existing blood pressure medication may slow the progression of a rare and fatal brain disease in children, according to a study by the Amsterdam University Medical Centers (Amsterdam UMC) in the Netherlands.

The study, published in the latest issue of The Lancet Neurology, examined the effects of guanabenches on vanishing white matter (VWM), a rare hereditary neurodegenerative disorder that affects children between the ages of 1 and 6 years.

VWM causes a gradual loss of motor and intellectual abilities and can lead to premature death. There is currently no approved treatment that can stop or slow the progression of the disease, according to the Amsterdam UMC.

Researchers followed children with VWM who received guanabenz and compared their disease progression over three years with the disease progression of 66 children with similar disease severity from an international registry who did not receive the drug, Xinhua reported.

They found that children treated with guanabenz became dependent on wheelchairs less frequently and less quickly than those in the comparison group.

“This shows for the first time that children can be affected by this fatal brain disease,” said Marjo van der Knaap, first author of the study and retired professor of pediatric neurology at the Medical University of Amsterdam.

According to the study, none of the children treated with guanabenze died during the study period, compared to five of 66 children in the comparison group.

Side effects, including hallucinations, drowsiness, constipation, and low blood pressure, occurred mainly during the first few months of treatment. After four to six months, the children generally tolerated the medication well, and none discontinued treatment due to side effects.

“Because we are specifically dealing with young children, it is important that the side effects are known, treatable and temporary,” van der Knaap said.

The researchers stressed that guanabenze is not a cure for VWM and that its beneficial effects appear to disappear after treatment is discontinued. They also cautioned that the study did not include an untreated control group at one time.

A follow-up study is underway to monitor children over a longer period and investigate the effects of high doses of guanabenze, according to the Amsterdam UMC.

VWM is extremely rare, with an estimated one in every 100,000 babies born with the disease worldwide. In the Netherlands, about 1.3 people per million live with this condition.